10:51am Friday 06 December 2019

Major new lead for Parkinson´s treatment

The study, primarily funded by the Parkinson´s Disease Society and published online by the journal Nature Neuroscience this week, identified a pathway inside nerve cells that could be stimulated to protect the dying cells affected by Parkinson´s.

Parkinson’s is caused by the progressive death of specific nerve cells inside the brain that produce dopamine – a chemical messenger that controls the co-ordination of movement.

Using both fruit fly models and skin cells from people with Parkinson´s, the researchers identified a common pathway inside the cells that can be stimulated to prevent cell death in inherited forms of the condition. Pathways are control systems which operate inside the cell that regulate all aspects of cell functions including determining whether they live or die. Finding new drugs that can interfere with these means that we could target these pathways and essentially halt, or even prevent, the death of the cells.

In the study the drug Rapamycin – used by some transplant patients to prevent immune rejection – was shown to protect cells against the damaging effects of two of the mutant genes that cause inherited forms of Parkinson´s.

Rapamycin is a potent immunosuppressant and comes with the serious risk of a weakened immune system, so is very unlikely to be used to treat Parkinson´s directly. However, investigating how this particular drug helps protects against cell death will help find more sophisticated ways to protect nerve cells without the unwanted side effects.

Dr Alex Whitworth, who led the research team in this study at the University of Sheffield, said: “Although Rapamycin is not a `wonder drug´ for treating Parkinson´s, our study does shows that the animal and human models that we used can be useful tool in the discovery of new drugs for directly treating the condition.

“Another exciting outcome of our study is that the positive effects were seen in both flies and human cells. This shows that even simple animal models do work in some cases, and that human cells grown in the lab may be a good method of screening for new anti-Parkinson´s drugs in the future.”

Dr Kieran Breen, Director of Research and Development at the Parkinson´s Disease Society, said: “This is an exciting new development in the search for new and better treatments for Parkinson´s. Current treatments can only replace or mimic the effects of dopamine, rather than actually change the course of the condition.

“But the discovery of this pathway may be the key to developing new drugs that can slow or even halt the progressive loss of nerve cells in the brain. Effectively, this would halt the development of Parkinson´s in its tracks.

“At the Parkinson´s Disease Society we are passionate about finding a cure and better treatments for people with Parkinson´s and these new findings are a major step forward for this goal.”

Notes for Editors: Fruit flies with inherited genes for Parkinson´s develop symptoms similar to people with Parkinson´s. The dopamine-producing nerve cells die and the flies have difficulty with co-ordinating movements for flying and walking. Giving Rapamycin to these flies prevented them from developing the Parkinson´s-like symptoms.

The researchers also looked at the effect of Rapamycin on skin cells taken from people with inherited Parkinson´s. In people with the mutation, the tiny, energy-producing batteries inside cells, known as mitochondria, do not work properly. But Rapamycin was able to ease these mitochondrial problems suggesting that the results we saw in fruit flies are relevant to real people with Parkinson´s.

The article will appear in print in the September issue of Nature Neuroscience, but a copy of the paper is available now.

The research was led by Dr Alex Whitworth as a collaboration between two different Parkinson´s Disease Society funded research groups at the University of Sheffield´s Department of Biomedical Science, who have received £417,845 in research grants between them. The Wellcome Trust also provided funding for this research.

The Parkinson´s Disease Society is the leading authority in the UK on the condition and a world leader in research, with over £40million invested in research over the last 40 years. We are closer than ever to finding a cure. We campaign for a better quality of life, provide expert information on all aspects of Parkinson´s and a local support network for people with Parkinson´s, their carers, families and friends. We are totally dependent on voluntary donations.

For further information please contact: Shemina Davis, Media Relations Officer, on 0114 2225339


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